Biotech Roundup: Duchenne Delay, Forum’s Fate, Califf, Ionis & More

Memorial Day weekend may be fast approaching, but don’t fire up those grills yet: there’s plenty of headlines to dig into first in our biotech trip around the Xconomy network.

In New York, the nation’s most prestigious science competition changed sponsors for just the second time in its 74-year history. In Boston, one company’s controversial quest to potentially get the first drug for Duchenne muscular dystrophy approved drags on, while the fate of another seems to hang by a thread.

And on the West Coast, the first implantable device for opioid addiction was approved—just as the FDA’s commissioner made some pointed remarks at a Stanford University chat about the future rate of drug approvals. Those stories and much more below.

—The multi-year saga of eteplirsen, a Duchenne muscular dystrophy drug from Cambridge, MA-based Sarepta Therapeutics—which could be the first ever approved for the deadly disease—will continue to drag on. The FDA said this week that it wouldn’t complete its view of the drug by the May 26 deadline, and didn’t give any timeline for when a decision would come. Shares of Sarepta (NASDAQ: SRPT) surged more than 25 percent as investors took the delay as a positive sign for eteplirsen’s approval chances. Forbes has more on the potential outcomes that might lie ahead.

—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) has been named the new sponsor of the nation’s oldest science competition, the “Science Talent Search” (formerly known as the Westinghouse Award), taking over for Intel, which gave up its sponsorship last year. Regeneron has pledged $100 million in a 10-year commitment to the competition. Here’s more from The New York Times.

—Watertown, MA-based Selecta Biosciences filed for an IPO this week and aims to trade on the Nasdaq under the ticker symbol “SELB.” Selecta is using nanotechnology to trick the immune system into calling off an unwanted attack on a particular target; its lead drug is for chronic refractory gout.

—Shares of Burlington, MA-based Flexion Therapeutics (NASDAQ: FLXN) climbed 50 percent as the FDA, according to the company, indicated that Flexion’s data for long-acting steroid injection FX006 are good enough to allow it to file an approval application.

—Theranos of Palo Alto, CA, is already in hot water with the DOJ, the SEC, and the folks who oversee Medicare. Now here come the personal injury lawyers after Theranos revealed last week it had to correct two years worth of blood test results. One lawsuit was filed in Northern California on behalf of an Arizona man, as the San Francisco Business Times reported.

—Shares of Carlsbad, CA-based Ionis Pharmaceuticals (NASDAQ: IONS) plunged by nearly 40 percent—eviscerating nearly $1 billion in market value—after partner GlaxoSmithKline decided to delay starting a Phase 3 study of its experimental RNA interference drug IONIS-TTRrx due to safety concerns. The FDA placed a clinical hold on a separate IONIS-TTRrx trial a few weeks ago, and according to a research note from Leerink Partners analyst Michael Schmidt, the company said on a conference call with analysts that cases of severe thrombocytopenia—dangerously low platelet counts—have been seen in the testing of two different Ionis treatments.

—Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) meanwhile, which has a rival drug to IONIS-TTRx—both are treatments for a rare condition called transthyretin amyloidosis—saw its shares jump 12 percent.

—Sanofi is trying to shake up San Francisco biotech Medivation (NASDAQ: MDVN), too. The French pharma is one of a few suitors, so far unwanted, for the co-owner of prostate cancer drug enzalutamide (Xtandi). Sanofi this week took its bid hostile, aiming to oust some Medivation boardmembers. Medivation urged shareholders to reject Sanofi’s efforts.

—Avalon Ventures and GSK formed their eighth San Diego biotech—a cancer drug developer called PDI Therapeutics—under a collaboration established three years ago. As with the other startups in the Avalon/GSK partnership, PDI will get up to $10 million in Series A cash and research funding, and GSK has an exclusive option to buy the startup later on.

​—Struggling San Diego-based Sophiris ​Bio ​(NASDAQ: SPHS) has hired an investment banking firm to help evaluate strategic alternatives to advance its experimental prostate drug. Sophiris also cut its workforce ​from 10 to five, and has raised $4.6 million to extend its operations for at least one more year.

—An unexpected $70.5 million investment in Tribune Publishing from biotech billionaire Patrick Soon-Shiong has apparently thwarted Gannett’s hostile buyout bid for the media giant, which owns the The Los Angeles Times and The San Diego Union-Tribune. Soon-Shiong’s Culver City, CA-based firm NantHealth, meanwhile, is expected to go public next week.